ENCePP Guide on Methodological Standards in Pharmacoepidemiology

10.1.1. Introduction

Comparative effectiveness
research (CER) is designed to inform health-care decisions at the level of both
policy and the individual by comparing the benefits and harms of therapeutic
strategies available in routine practice, for the prevention, the diagnosis or
the treatment of a given health condition. The interventions under comparison
may be related to similar treatments, such as competing drugs, or different
approaches, such as surgical procedures and drug therapy. The comparison may
focus only on the relative medical benefits and risks of the different options
or it may weigh both their costs and their benefits. The methods of comparative effectiveness research (Annu
Rev Public Health 2012;33:425-45) defines the key elements of CER as (a)
head-to-head comparison of active treatments, (b) study populations typical of
day-to-day clinical practice, and (c) a focus on evidence to inform health care
tailored to the characteristics of individual patients. In What is Comparative Effectiveness Research, the AHRQ
highlights that CER requires the development, expansion and use of a variety of
data sources and methods to conduct timely and relevant research and disseminate
the results in a form that is quickly usable. The evidence may come from a
review and synthesis of available evidence from existing clinical trials or
observational studies or from the conduct of studies that generate new evidence.
In Developing a Protocol for Observational Comparative Effectiveness
Research: A User’s Guide, AHRQ also highlights that CER is still a relatively new
field of enquiry that has its origin across multiple disciplines and is likely
to evolve and be refined over time.

Among resources for keeping up
with the evolution in this field, the US National Library of Medicine provides a
web site for queries on
CER.